Our Research
Overview
Ryvu Therapeutics discovers and develops small molecule therapies that address high value emerging targets in oncology.
Our pipeline is built from internally-discovered candidates which make use of diverse therapeutic mechanisms, including programs directed at targets in the areas of transcriptional regulation, synthetic lethality and immuno-oncology.
Ryvu’s two most advanced programs are currently in clinical trials. RVU120 is a selective CDK8 kinase inhibitor in clinical development for the treatment of acute myeloid leukemia and high-risk myelodysplastic syndromes, as well as solid tumors.
SEL24 (MEN1703) is a dual PIM/FLT3 kinase inhibitor in clinical development for the treatment of acute myeloid leukemia. Additional programs include synthetic lethality candidates directed at WRN and MTAP deletions as well as immuno-oncology programs focused on STING agonists and HPK1.
Clinical Projects
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RVU120
CDK8 is a kinase submodule of the mediator complex, involved in both transcriptional activation and repression. CDK8- mediator complex integrates basal transcriptional machinery with the activity of oncogenic transcriptional and epigenetic factors. Targeting CDK8 and...
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SEL24 (MEN1703)
SEL24 (MEN1703) is a selective, dual inhibitor of PIM and FLT3 kinases, two enzymes that are strongly implicated in malignant transformation of hematopoietic cells. The compound is a novel small molecule discovered by Ryvu and...
Immuno-oncology
Ryvu Therapeutics has deep expertise in the discovery of small molecule candidates with optimized properties to address specific limitations of existing therapeutic approaches.
To identify these candidates, Ryvu uses platforms based on the most current understanding of how the immune system interacts with cancer cells and how cancer cells function biologically. These immuno-oncology and immunometabolism approaches have a successful history in biopharmaceutical development. Ryvu has accumulated scientific and technical infrastructure that allow high-throughput generation of candidates using these platforms.

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STING AGONIST
STING protein is one of the major players in innate immune activation being a potent trigger of type I interferon (IFN) and pro-inflammatory cytokines. STING agonists facilitate cancer antigen recognition specific to individual patient and...
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HPK1 INHIBITORS
The high therapeutic potential of HPK1 inhibitors (hematopoietic progenitor kinase 1, MAP4K1) lies in their potential to simultaneously improve T cell function, tumor antigen presentation and combat the immunosuppressive tumor microenvironment. HPK1 is a negative...
Synthetic Lethality
One of the challenges in the development of new oncology therapeutics is the limited number of protein targets known to be addressable with small molecules. Synthetic lethality allows the identification of novel protein targets and mechanisms of cancer cell sensitivity that can be used therapeutically.
Compounds active on specific synthetic lethal targets selectively kill tumor cells of a precisely defined model system, characterized by a specific genetic, epigenetic or metabolic characteristic. This is the approach actively investigated at Ryvu that allows the generation of compounds with highly specific and well-characterized activity.

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MTA-COOPERATIVE PRMT5 INHIBITOR
Deletion of 9p21 locus is one of the most frequent genetic alterations associated with human cancers. Apart from tumour suppressor CDKN2A, locus 9p21 contains the gene coding methylthioadenosine phosphorylase (MTAP), the only enzyme capable of...
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WRN INHIBITOR
Werner Syndrome helicase (WRN) is a member of the RecQ helicase family and plays an important role in controlling DNA repair mechanisms and maintaining integrity of the genome. WRN helicase has been identified to be...
Novel Targets
Beyond publicly disclosed programs, Ryvu is working on candidates that address novel targets in the areas of synthetic lethality, immuno-oncology and immunometabolism to generate new therapeutics with first-in-class or best-in-class potential.
For the candidates, Ryvu is utilizing a proprietary drug discovery approach combining internally developed expertise and new sophisticated tools and technologies to identify small molecule compounds with favorable profiles and activity.
Discovery Collaborations

In November 2022, Ryvu Therapeutics and BioNTech have entered into a multi-target research collaboration for several small molecule immunotherapy programs. BioNTech and Ryvu will jointly undertake drug discovery and research projects to develop multiple small molecule programs directed at exclusive targets selected by BioNTech, primarily focused on immune modulation within oncology, with potential applications in other disease areas.

In April 2020, Ryvu Therapeutics and Galapagos have entered into a collaboration focused on the discovery and development of novel small molecule drugs in inflammation.
The collaboration is based on a novel drug target identified by Ryvu, which will contribute its technology platform and related intellectual property. Ryvu and Galapagos will both provide resources to support the collaboration and make use of their expertise in high-throughput screening, biology, medicinal chemistry, and toxicology.
During the joint research collaboration, Ryvu is responsible for early drug discovery and Galapagos will be responsible for all further development of the program.

In 2013, Ryvu and Merck KGaA initiated a joint research strategy involving intensive work on multiple targets and several chemical series. The collaboration has successfully completed two milestones and has been expanded over time with additional targets.
The aim of the partnership with Merck is the development of new anticancer therapeutics acting on diverse biological targets associated with aberrant metabolic pathways in cancer cells. Dependence on specific metabolic pathways is a feature of various types of cancer, therefore, targeting this vulnerability has many potential applications in a broad range of patient populations.
The collaboration has resulted in scientific achievements, the joint filing of patent applications and data presentations at international scientific conferences.
Publications
American Society of Hematology (ASH) Annual Meeting, December 10 –13, 2022
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CDK8/19 Kinase Inhibitor RVU120 in Patients with AML or Higher-Risk MDS: Safety and Efficacy Results from New Dose Escalation Cohorts
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Multiomics Analysis Confirms Effective Target Engagement for RVU120 – a First-in-class CDK8/19 Kinase Inhibitor in AML and MR-MDS Patients and Reveals the Mechanism of Action
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PIM Inhibition by SEL24 (MEN1703) Combines Synergistically with gilteritinib in FLT3-ITD Preclinical Models of Acute Myeloid Leukemia
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Super-enhancer-driven PIM Kinase Upregulation in Multiple Myeloma Maintains the Plasma Cell-specific Oncogenic and Microenvironmental Circuits and Can Be Efficiently Targeted by the Pan-PIM Inhibitor MEN1703
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PIM Kinases Regulate Super-Enhancer-Dependent Gene Expression In Diffuse Large B-Cell Lymphoma
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MEN1703-mediated PIM kinases inhibition impairs protumoral and immunosuppressive phenotype and functions of macrophages in classical Hodgkin Lymphoma
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EORTC-NCI-AACR Symposium (ENA), October 26-28, 2022
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Phase I/II trial of RVU120, a CDK8/CDK19 inhibitor in patients with relapsed/refractory metastatic or advanced solid tumors
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RVU120, a small molecule inhibitor of CDK8/19 kinases, enhances rituximab-driven NK cells-mediated cytotoxicity both in vitro and in vivo
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Discovery of novel MTA-cooperative PRMT5 inhibitors as targeted therapeutics for MTAP deleted cancers
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European Hematology Association (EHA) Hybrid Congress, June 9-17, 2022
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CLI120-001 Phase1b Dose Escalation Study of RVU120 in Patients with AML or High-Risk MDS Safety and Efficacy Data Update
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Preclinical and Clinical Signs of RVU120 efficacy, a Specific CDK8/19 Inhibitor in DNMT3A and NPM1 Mutation Positive AML and HR-MDS
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Phase 1/2 Study of SEL24/MEN1703, a First-In-Class Dual PIM/FLT3 Kinase Inhibitor, in Patients with IDH1/2-Mutated Acute Myeloid Leukemia: the DIAMOND-01 Trial
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American Society of Clinical Oncology (ASCO) Annual Meeting, June 3-7, 2022
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Phase 1/2 study of SEL24/MEN1703, a first-in-class dual PIM/FLT3 kinase inhibitor, in patients with IDH1/2-mutated acute myeloid leukemia: the DIAMOND-01 trial
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Phase I/II trial of RVU120 (SEL120), a CDK8/CDK19 inhibitor in patients with relapsed/refractory metastatic or advanced solid tumors